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Development of anti-DUX4 therapeutics for FSHD

Posted by George Shaw on September 6, 2012

Dr. Michael Kyba, Ph.D.

This grant was supplementary support linked to a National Institutes of Health (USA) NIH R21 (2-year) grant directed towards identifying chemical drugs that inhibit the genetic material (gene) called DUX4, which has been associated with causing FSHD. Dr Kyba’s group had previously screened 200,000 compounds and identified approximately 600 drugs inhibiting the cell death induced by the DUX4. Their current work is directed towards identifying the most promising drugs for treatment of FSHD, within this set of 600. Using the support from the Friends of FSH Research, they have performed additional screens on panels of chemicals known to be active in treating other diseases and in doing so identified several additional drugs inhibiting the DUX4-toxicity. The chemicals Dr Kyba’s group has identified may help us to understand the chemical pathways that can indirectly inhibit the muscle toxicity associated with the DUX4 gene. In the past year, they have obtained approximately 100 chemicals from the set of 600 identified, for further testing. The majority of these 600 chemicals fall into 20 different groups, known as “chemical series”. Members of these chemical series have similar chemical structures. Dr Kyba’s group repeated the initial screening of these chemicals, for protection of cells from DUX4-induced death, using the independently obtained compounds and they have now begun additional tests to understand how these compounds work and what chemical pathways they impact. Their objective now is to narrow down this set of 20 chemical series to identify the 2-4 that are most suitable for drug development for effective treatment of FSHD.

— Michael Kyba

This funding supplements an R21 grant Michael Kyba, an Associate Professor at the Lillehei Heart Institute, was recently awarded by NIH/NINDS. The 2-year grant allows additional resources to be put on this project in order to move it forward quickly. The project is being done in collaboration with a medicinal chemist at the Institute for Therapeutics Discovery and Development at the University of Minnesota, an investigator with experience at bringing drugs from the current stage all the way to clinical trials.